SpliceBio is a genetic medicines company that leverages its proprietary Protein Splicing platform to advance the development of next-generation gene therapies. This innovative platform is rooted in over two decades of research in intein and protein engineering conducted at Princeton University. By utilizing this technology, SpliceBio aims to address genetic diseases, with a particular focus on inherited retinal disorders. The company has recently entered into a collaboration with Spark Therapeutics to develop a gene therapy targeting an undisclosed inherited retinal disease, showcasing the potential applications of its Protein Splicing technology in clinical settings.

The company’s lead program targets Stargardt disease, the most prevalent inherited retinal disorder linked to mutations in the ABCA4 gene. Given the size of this gene, which is too large for conventional single AAV vectors, SpliceBio's Protein Splicing platform offers a unique solution for delivering effective gene therapies. In addition to Stargardt disease, SpliceBio is exploring various other therapeutic areas, indicating the versatility and broad applicability of its technology in treating genetic conditions. This strategic focus on diverse gene therapy programs underscores SpliceBio's commitment to pioneering advancements in the field of genetic medicine.

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SpliceBio is a biotechnology company exploiting Protein Splicing to develop the next generation of gene therapies. Our proprietary platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein and protein engineering research by our co-founders. The company is backed by UCB Ventures, Ysios Capital, NEA, Gilde Healthcare, Novartis Venture Fund and Asabys Partners.

SpliceBio

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